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Evaluation of the Tolerance and Efficiency of a Combined Oral Therapy With Lithium and GTA in Patients With Canavan Disease
The aim of this study is to determine whether oral supplementation with lithium and acetate may improve the biological and clinical prognosis in patients with Canavan Disease.
Details
| Lead sponsor | Assistance Publique - Hôpitaux de Paris |
|---|---|
| Phase | Phase 2 |
| Status | WITHDRAWN |
| Start date | 2009-09 |
| Completion | 2011-01 |
Conditions
- Canavan Disease
- Infantile
- Deficiency Disease
- Aspartoacylase
- Leukodystrophy, Spongiform
Interventions
- Lithium Gluconate (drug) Glyceryl Triacetate GTA (drug)
Primary outcomes
- The primary outcome will be a decrease of the NAA peak (> 20%) or the appearance of an acetate peak at the end of the treatment (10 months), using spectroscopy-MRI. — 10 months